Cost-effectiveness analysis of an orphan drug tebentafusp in patients with metastatic uveal melanoma and a call for value-based pricing.

IF 1.5 4区医学 Q3 DERMATOLOGY

Melanoma Research Pub Date : 2023-12-01 Epub Date: 2023-08-25 DOI:10.1097/CMR.0000000000000919

Shaohong Luo, Chen Xie, Ningning Lin, Dong Lin, Dian Gu, Shen Lin, Xiaoting Huang, Xiongwei Xu, Xiuhua Weng

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Abstract

The normative regimens recommendations for treating metastatic uveal melanoma (mUM) are absent in the US. Recently, a phase III randomized clinical trial revealed that tebentafusp yielded a conspicuously longer overall survival than the control group. Based on the prominent efficacy, this study aimed to assess whether tebentafusp is cost-effective compared to the control group in patients with untreated mUM. A three-state partitioned survival model was developed to assess the costs, quality-adjusted life years (QALYs), and incremental cost-effectiveness ratio (ICER) from the perspective of US payers. Scenario analyses and sensitivity analyses were conducted to explore the conclusion uncertainty. Compared with control group, tebentafusp therapy yielded an additional 0.47 QALYs (1.19 vs. 0.72 QALYs) and an incremental cost of $444 280 ($633 822 vs. $189 542). The resultant ICER of $953 230/QALY far outweighed the willingness-to-pay threshold of $200 000/QALY. The ICER was always more than $750 000/QALY in all the univariable and probabilistic sensitivity analyses. Scenario analyses indicated that reducing the unit price of tebentafusp to $33.768/µg was associated with a favorable result of tebentafusp being cost-effective. For treatment-naive patients with mUM, the cost of tebentafusp therapy was not worth the improvement in survival benefits at the current price compared to the investigator's choice of therapy. The cost-effectiveness of tebentafusp could be promoted using value-based pricing.

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孤儿药物tebentafusp治疗转移性葡萄膜黑色素瘤患者的成本效益分析和基于价值的定价呼吁。

美国缺乏治疗转移性葡萄膜黑色素瘤（mUM）的规范方案建议。最近，一项III期随机临床试验显示，tebentafusp的总生存期明显长于对照组。基于显著的疗效，本研究旨在评估在未经治疗的mUM患者中，与对照组相比，tebentafusp是否具有成本效益。开发了一个三州分割生存模型，从美国支付者的角度评估成本、质量调整寿命（QALYs）和增量成本效益比（ICER）。对结论的不确定性进行了情景分析和敏感性分析。与对照组相比，tebentafusp治疗产生了0.47个额外的QALYs（1.19对0.72个QALYs）和444美元的增量成本 280（633美元 822美元对189美元 542）。由此产生的953美元的ICER 230/QALY远远超过了200美元的支付门槛 000/QALY。ICER总是超过750美元 000/QALY。情景分析表明，将tebentafusp的单价降至33.768/µg与tebentausp具有成本效益的有利结果有关。对于未接受治疗的mUM患者，与研究者的治疗选择相比，以目前的价格计算，替本他福普治疗的成本不值得提高生存效益。可以使用基于价值的定价来提高tebentafusp的成本效益。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Melanoma Research 医学-皮肤病学

CiteScore

3.40

自引率

4.50%

发文量

139

审稿时长

6-12 weeks

期刊介绍： Melanoma Research is a well established international forum for the dissemination of new findings relating to melanoma. The aim of the Journal is to promote the level of informational exchange between those engaged in the field. Melanoma Research aims to encourage an informed and balanced view of experimental and clinical research and extend and stimulate communication and exchange of knowledge between investigators with differing areas of expertise. This will foster the development of translational research. The reporting of new clinical results and the effect and toxicity of new therapeutic agents and immunotherapy will be given emphasis by rapid publication of Short Communications. Thus, Melanoma Research seeks to present a coherent and up-to-date account of all aspects of investigations pertinent to melanoma. Consequently the scope of the Journal is broad, embracing the entire range of studies from fundamental and applied research in such subject areas as genetics, molecular biology, biochemistry, cell biology, photobiology, pathology, immunology, and advances in clinical oncology influencing the prevention, diagnosis and treatment of melanoma.